Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
This multicenter, open-label study is designed to assess safety, dose-response using pharmacokinetic (PK) and pharmacodynamic (PD) measures, and clinical efficacy of BMN 110 in subjects between 5 and 18 years of age, diagnosed with Mucopolysaccharidosis IVA (MPS IVA).
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| BMN 110 | Experimental | Within-patient Dose-Escalation |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| BMN 110 | Drug | Subjects will receive a weekly 4- to 5-hour intravenous infusion of BMN 110 in 3 consecutive 12-week dosing intervals, using the following regimen:
Subjects who complete the 36-week Dose-Escalation Period will have the option to continue drug treatment for an additional 36 to 48 weeks. Subjects continuing on treatment after the Dose-Escalation period will receive weekly 4- to 5-hour intravenous infusions of BMN 110 at a dose of 1.0 mg/kg/week. |
| Measure | Description | Time Frame |
|---|---|---|
| Subject Incidence of Treatment Emergent AEs | The primary objective of the study was to evaluate the safety of weekly infusions of BMN 110 administered in escalating doses to subjects with MPS IVA. The safety variable incidence of TEAE is summarized. | Entire Study, through week 84 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in 6MWT | Change from baseline in meters in 6-minute Walk Test. As a measure of endurance, a 6-minute walk test (6MWT) was performed according to the American Thoracic Society Guidelines. Patients were instructed to walk as far as possible in 6 minutes. | Baseline to Weeks 12, 24, 36, 48, 72 |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Celeste Decker, MD | BioMarin Pharmceutical Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Birmingham | United Kingdom | |||||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 29526614 | Derived | Hendriksz C, Santra S, Jones SA, Geberhiwot T, Jesaitis L, Long B, Qi Y, Hawley SM, Decker C. Safety, immunogenicity, and clinical outcomes in patients with Morquio A syndrome participating in 2 sequential open-label studies of elosulfase alfa enzyme replacement therapy (MOR-002/MOR-100), representing 5 years of treatment. Mol Genet Metab. 2018 Apr;123(4):479-487. doi: 10.1016/j.ymgme.2018.02.011. Epub 2018 Feb 19. |
| Label | URL |
|---|---|
| BioMarin Pharmaceutical Inc. website | View source |
Not provided
Not provided
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | BMN 110 | Dose-Escalation Period: Subjects will receive a weekly 4- to 5-hour intravenous infusion of BMN 110 in 3 consecutive 12-week dosing intervals, using the following regimen:
Continuation Period: Subjects who complete the 36-week Dose-Escalation Period will have the option to continue drug treatment for an additional 36 to 48 weeks. Subjects continuing on treatment after the Dose-Escalation period will receive weekly 4- to 5-hour intravenous infusions of BMN 110 at a dose of 1.0 mg/kg/week. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Weeks 1-12: 0.1 mg/kg/Week |
|
| |||||||||||||||||||||
| Weeks 13-24: 1.0 mg/kg/Week |
| ||||||||||||||||||||||
| Weeks 25-36: 2.0 mg/kg/Week |
| ||||||||||||||||||||||
| Continuation Period |
|
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | BMN 110 | Dose-Escalation Period: |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Secondary | Change From Baseline in 6MWT | Change from baseline in meters in 6-minute Walk Test. As a measure of endurance, a 6-minute walk test (6MWT) was performed according to the American Thoracic Society Guidelines. Patients were instructed to walk as far as possible in 6 minutes. | Intent-to-Treat population (all subjects who enrolled in the study). Two patients were either physically (score was designated as 0 m) or developmentally (score was set to missing) unable to perform the 6MWT. The analysis was based on observed cases. | Posted | Mean | Standard Deviation | meters | Baseline to Weeks 12, 24, 36, 48, 72 |
|
Study Period, through 84 weeks
All Adverse Events
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 0.1 mg/kg/Week | Dose-Escalation Period: Weeks 1-12: 0.1 mg/kg/week Subjects will receive a weekly 4- to 5-hour intravenous infusion of BMN 110 0.1 mg/kg/week |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Gait disturbance | General disorders | MedDRA 12.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA 12.0 | Systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| BioMarin Medical Information Services | BioMarin Pharmaceutical Inc. | 800-983-4587 | medinfo@bmrn.com |
Not provided
| ID | Term |
|---|---|
| D009085 | Mucopolysaccharidosis IV |
| D016464 | Lysosomal Storage Diseases |
| ID | Term |
|---|---|
| D009083 | Mucopolysaccharidoses |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
Not provided
Not provided
| ID | Term |
|---|---|
| C102295 | GALNS protein, human |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
|
| Change From Baseline in 3MSCT |
Change from baseline in the 3-minute Stair Climb Test. Patients walked up stairs that have a railing, which could be used for support, for 3 minutes, with the number of stairs climbed recorded. The test result was the number of steps climbed per minute. |
| Baseline to Weeks 12, 24, 36, 48, 72 |
| Percent Change From Baseline in uKS | Percent Change from baseline in Normalized Urine KS. The percent change was calculated (Week X value - baseline value)/baseline value *100% | Baseline to Weeks 12, 24, 36, 72 |
| Percent Change From Baseline in MVV | Percent Change from baseline in Maximum Voluntary Ventilation. | Baseline to Weeks 12, 24, 36, 72 |
| Percent Change From Baseline in FVC | Percent Change from baseline in Forced Vital Capacity. | Baseline to Weeks 12, 24, 36, 72 |
| London |
| United Kingdom |
| Manchester | United Kingdom |
| years |
|
| Age, Customized | Number | participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Number | participants |
|
| Region of Enrollment | Number | participants |
|
|
|
| Secondary | Change From Baseline in 3MSCT | Change from baseline in the 3-minute Stair Climb Test. Patients walked up stairs that have a railing, which could be used for support, for 3 minutes, with the number of stairs climbed recorded. The test result was the number of steps climbed per minute. | Intent-to-Treat population (all subjects who enrolled in the study). One patient was developmentally unable to perform the 3MSCT and the test scores were set to missing. The analysis was based on observed cases. | Posted | Mean | Standard Deviation | steps/min | Baseline to Weeks 12, 24, 36, 48, 72 |
|
|
|
| Secondary | Percent Change From Baseline in uKS | Percent Change from baseline in Normalized Urine KS. The percent change was calculated (Week X value - baseline value)/baseline value *100% | Intent-to-Treat population (all subjects who enrolled in the study). The analysis was based on observed cases. | Posted | Mean | Standard Deviation | percentage of uKS | Baseline to Weeks 12, 24, 36, 72 |
|
|
|
| Secondary | Percent Change From Baseline in MVV | Percent Change from baseline in Maximum Voluntary Ventilation. | Intent-to-Treat population (all subjects who enrolled in the study). The analysis was based on observed cases. | Posted | Mean | Standard Deviation | percentage of MVV | Baseline to Weeks 12, 24, 36, 72 |
|
|
|
| Secondary | Percent Change From Baseline in FVC | Percent Change from baseline in Forced Vital Capacity. | Intent-to-Treat population (all subjects who enrolled in the study). The analysis was based on observed cases. | Posted | Mean | Standard Deviation | percentage of FVC | Baseline to Weeks 12, 24, 36, 72 |
|
|
|
| Primary | Subject Incidence of Treatment Emergent AEs | The primary objective of the study was to evaluate the safety of weekly infusions of BMN 110 administered in escalating doses to subjects with MPS IVA. The safety variable incidence of TEAE is summarized. | Posted | Number | participants | Entire Study, through week 84 |
|
|
|
| 6 |
| 20 |
| 18 |
| 20 |
| EG001 | 1.0 mg/kg/Week | Dose-Escalation Period: Weeks 13-24: 1.0 mg/kg/week Subjects will receive a weekly 4- to 5-hour intravenous infusion of BMN 110 1.0 mg/kg/week | 2 | 18 | 18 | 18 |
| EG002 | 2.0 mg/kg/Week | Dose-Escalation Period: Weeks 25-36: 2.0 mg/kg/week Subjects will receive a weekly 4- to 5-hour intravenous infusion of BMN 110 2.0 mg/kg/week | 8 | 18 | 16 | 18 |
| EG003 | Continuation Period | Subjects who complete the 36-week Dose-Escalation Period will have the option to continue drug treatment for an additional 36 to 48 weeks. Subjects continuing on treatment after the Dose-Escalation period will receive weekly 4- to 5-hour intravenous infusions of BMN 110 at a dose of 1.0 mg/kg/week. | 6 | 18 | 17 | 18 |
| EG004 | Entire Study | Entire Study period includes both Dose-Escalation Period and Continuation Period. | 14 | 20 | 20 | 20 |
| Infusion related reaction | General disorders | MedDRA 12.0 | Systematic Assessment |
|
| Type I hypersensitivity | Immune system disorders | MedDRA 12.0 | Systematic Assessment |
|
| Abdominal abscess | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Abscess limb | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Catheter site infection | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Implant site infection | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Lower respiratory tract infection | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Otitis media | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Road traffic accident | Injury, poisoning and procedural complications | MedDRA 12.0 | Systematic Assessment |
|
| Knee deformity | Musculoskeletal and connective tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Drug eruption | Skin and subcutaneous tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Rash generalised | Skin and subcutaneous tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Abscess drainage | Surgical and medical procedures | MedDRA 12.0 | Systematic Assessment |
|
| Catheterisation venous | Surgical and medical procedures | MedDRA 12.0 | Systematic Assessment |
|
| Poor venous access | Vascular disorders | MedDRA 12.0 | Systematic Assessment |
|
| Mitral valve disease | Cardiac disorders | MedDRA 12.0 | Systematic Assessment |
|
| Tachycardia | Cardiac disorders | MedDRA 12.0 | Systematic Assessment |
|
| Cerumen impaction | Ear and labyrinth disorders | MedDRA 12.0 | Systematic Assessment |
|
| Deafness neurosensory | Ear and labyrinth disorders | MedDRA 12.0 | Systematic Assessment |
|
| Ear canal erythema | Ear and labyrinth disorders | MedDRA 12.0 | Systematic Assessment |
|
| Ear disorder | Ear and labyrinth disorders | MedDRA 12.0 | Systematic Assessment |
|
| Ear pain | Ear and labyrinth disorders | MedDRA 12.0 | Systematic Assessment |
|
| External ear disorder | Ear and labyrinth disorders | MedDRA 12.0 | Systematic Assessment |
|
| Hyperacusis | Ear and labyrinth disorders | MedDRA 12.0 | Systematic Assessment |
|
| Hypoacusis | Ear and labyrinth disorders | MedDRA 12.0 | Systematic Assessment |
|
| Inner ear disorder | Ear and labyrinth disorders | MedDRA 12.0 | Systematic Assessment |
|
| Motion sickness | Ear and labyrinth disorders | MedDRA 12.0 | Systematic Assessment |
|
| Otorrhoea | Ear and labyrinth disorders | MedDRA 12.0 | Systematic Assessment |
|
| Tinnitus | Ear and labyrinth disorders | MedDRA 12.0 | Systematic Assessment |
|
| Autoimmune thyroiditis | Endocrine disorders | MedDRA 12.0 | Systematic Assessment |
|
| Conjunctivitis | Eye disorders | MedDRA 12.0 | Systematic Assessment |
|
| Eye discharge | Eye disorders | MedDRA 12.0 | Systematic Assessment |
|
| Eyelid cyst | Eye disorders | MedDRA 12.0 | Systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Dental caries | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Faecal incontinence | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Glossodynia | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Retching | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Salivary gland enlargement | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Toothache | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Application site vesicles | General disorders | MedDRA 12.0 | Systematic Assessment |
|
| Catheter site erythema | General disorders | MedDRA 12.0 | Systematic Assessment |
|
| Catheter site pain | General disorders | MedDRA 12.0 | Systematic Assessment |
|
| Chills | General disorders | MedDRA 12.0 | Systematic Assessment |
|
| Extravasation | General disorders | MedDRA 12.0 | Systematic Assessment |
|
| Feeling hot | General disorders | MedDRA 12.0 | Systematic Assessment |
|
| Gait disturbance | General disorders | MedDRA 12.0 | Systematic Assessment |
|
| Implant site erythema | General disorders | MedDRA 12.0 | Systematic Assessment |
|
| Implant site extravasation | General disorders | MedDRA 12.0 | Systematic Assessment |
|
| Implant site rash | General disorders | MedDRA 12.0 | Systematic Assessment |
|
| Influenza like illness | General disorders | MedDRA 12.0 | Systematic Assessment |
|
| Infusion site erythema | General disorders | MedDRA 12.0 | Systematic Assessment |
|
| Infusion site inflammation | General disorders | MedDRA 12.0 | Systematic Assessment |
|
| Infusion site oedema | General disorders | MedDRA 12.0 | Systematic Assessment |
|
| Injection site pain | General disorders | MedDRA 12.0 | Systematic Assessment |
|
| Injection site reaction | General disorders | MedDRA 12.0 | Systematic Assessment |
|
| Malaise | General disorders | MedDRA 12.0 | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA 12.0 | Systematic Assessment |
|
| Hepatomegaly | Hepatobiliary disorders | MedDRA 12.0 | Systematic Assessment |
|
| Drug hypersensitivity | Immune system disorders | MedDRA 12.0 | Systematic Assessment |
|
| Hypersensitivity | Immune system disorders | MedDRA 12.0 | Systematic Assessment |
|
| Abdominal abscess | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Catheter site infection | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Ear infection | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Eye infection | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Gastroenteritis viral | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Helminthic infection | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Impetigo | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Infection | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Lice infestation | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Localised infection | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Lower respiratory tract infection | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Nail infection | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Otitis externa | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Pharyngitis | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Rhinitis | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Skin infection | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Subcutaneous abscess | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Tinea pedis | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Tonsillitis | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Varicella | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
|
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA 12.0 | Systematic Assessment |
|
| Arthropod sting | Injury, poisoning and procedural complications | MedDRA 12.0 | Systematic Assessment |
|
| Contusion | Injury, poisoning and procedural complications | MedDRA 12.0 | Systematic Assessment |
|
| Device migration | Injury, poisoning and procedural complications | MedDRA 12.0 | Systematic Assessment |
|
| Excoriation | Injury, poisoning and procedural complications | MedDRA 12.0 | Systematic Assessment |
|
| Eye injury | Injury, poisoning and procedural complications | MedDRA 12.0 | Systematic Assessment |
|
| Face injury | Injury, poisoning and procedural complications | MedDRA 12.0 | Systematic Assessment |
|
| Fall | Injury, poisoning and procedural complications | MedDRA 12.0 | Systematic Assessment |
|
| Head injury | Injury, poisoning and procedural complications | MedDRA 12.0 | Systematic Assessment |
|
| Injury | Injury, poisoning and procedural complications | MedDRA 12.0 | Systematic Assessment |
|
| Joint injury | Injury, poisoning and procedural complications | MedDRA 12.0 | Systematic Assessment |
|
| Medical device complication | Injury, poisoning and procedural complications | MedDRA 12.0 | Systematic Assessment |
|
| Mouth injury | Injury, poisoning and procedural complications | MedDRA 12.0 | Systematic Assessment |
|
| Procedural pain | Injury, poisoning and procedural complications | MedDRA 12.0 | Systematic Assessment |
|
| Procedural vomiting | Injury, poisoning and procedural complications | MedDRA 12.0 | Systematic Assessment |
|
| Thermal burn | Injury, poisoning and procedural complications | MedDRA 12.0 | Systematic Assessment |
|
| Alanine aminotransferase increased | Investigations | MedDRA 12.0 | Systematic Assessment |
|
| Aspartate aminotransferase increased | Investigations | MedDRA 12.0 | Systematic Assessment |
|
| Blood immunoglobulin E increased | Investigations | MedDRA 12.0 | Systematic Assessment |
|
| Blood immunoglobulin G decreased | Investigations | MedDRA 12.0 | Systematic Assessment |
|
| Blood lactate dehydrogenase increased | Investigations | MedDRA 12.0 | Systematic Assessment |
|
| Blood sodium increased | Investigations | MedDRA 12.0 | Systematic Assessment |
|
| Cardiac murmur | Investigations | MedDRA 12.0 | Systematic Assessment |
|
| Computerised tomogram | Investigations | MedDRA 12.0 | Systematic Assessment |
|
| Echocardiogram abnormal | Investigations | MedDRA 12.0 | Systematic Assessment |
|
| Electrocardiogram T wave amplitude decreased | Investigations | MedDRA 12.0 | Systematic Assessment |
|
| Eosinophil count increased | Investigations | MedDRA 12.0 | Systematic Assessment |
|
| Nuclear magnetic resonance imaging | Investigations | MedDRA 12.0 | Systematic Assessment |
|
| Oxygen saturation decreased | Investigations | MedDRA 12.0 | Systematic Assessment |
|
| Protein total abnormal | Investigations | MedDRA 12.0 | Systematic Assessment |
|
| Respiratory rate increased | Investigations | MedDRA 12.0 | Systematic Assessment |
|
| Weight increased | Investigations | MedDRA 12.0 | Systematic Assessment |
|
| Dehydration | Metabolism and nutrition disorders | MedDRA 12.0 | Systematic Assessment |
|
| Hypercholesterolaemia | Metabolism and nutrition disorders | MedDRA 12.0 | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Bursitis | Musculoskeletal and connective tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Mobility decreased | Musculoskeletal and connective tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Pain in jaw | Musculoskeletal and connective tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Ageusia | Nervous system disorders | MedDRA 12.0 | Systematic Assessment |
|
| Clonus | Nervous system disorders | MedDRA 12.0 | Systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA 12.0 | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA 12.0 | Systematic Assessment |
|
| Lethargy | Nervous system disorders | MedDRA 12.0 | Systematic Assessment |
|
| Spinal cord compression | Nervous system disorders | MedDRA 12.0 | Systematic Assessment |
|
| Insomnia | Psychiatric disorders | MedDRA 12.0 | Systematic Assessment |
|
| Enuresis | Renal and urinary disorders | MedDRA 12.0 | Systematic Assessment |
|
| Incontinence | Renal and urinary disorders | MedDRA 12.0 | Systematic Assessment |
|
| Urinary incontinence | Renal and urinary disorders | MedDRA 12.0 | Systematic Assessment |
|
| Balanitis | Reproductive system and breast disorders | MedDRA 12.0 | Systematic Assessment |
|
| Penile pain | Reproductive system and breast disorders | MedDRA 12.0 | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Dry throat | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Pharyngeal oedema | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Rales | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Dermatitis diaper | Skin and subcutaneous tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Petechiae | Skin and subcutaneous tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Psoriasis | Skin and subcutaneous tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Rash generalised | Skin and subcutaneous tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Rash papular | Skin and subcutaneous tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Rash pruritic | Skin and subcutaneous tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Skin disorder | Skin and subcutaneous tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Skin lesion | Skin and subcutaneous tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Skin ulcer | Skin and subcutaneous tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Cautery to nose | Surgical and medical procedures | MedDRA 12.0 | Systematic Assessment |
|
| Induction of anaesthesia | Surgical and medical procedures | MedDRA 12.0 | Systematic Assessment |
|
| Flushing | Vascular disorders | MedDRA 12.0 | Systematic Assessment |
|
| Hot flush | Vascular disorders | MedDRA 12.0 | Systematic Assessment |
|
| Hypotension | Vascular disorders | MedDRA 12.0 | Systematic Assessment |
|
| Poor venous access | Vascular disorders | MedDRA 12.0 | Systematic Assessment |
|
PI cannot publish any data prior to multi-centre publication. If publication related to sub-sets of data shall make reference to the relevant multi-centre publication. PI agrees to submit data to Sponsor for review and comment 60 days prior to publication. During review period, Sponsor may request that publication be delayed for up to 6 months from date of first submission to Sponsor in order for Sponsor to take steps to protect its proprietary information.
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
|
| Week 48 Change from Baseline (n=17) |
|
| Week 72 Change from Baseline (n=17) |
|
|
| Week 72 Percent Change from Baseline (n=17) |
|
|
| Week 72 Percent Change from Baseline (n=14) |
|
|
| Week 72 Percent Change from Baseline (n=16) |
|
| Any Study Drug-Related AEs |
|
| Any SAEs |
|
| Any Study Drug-Related SAEs |
|
| Any AEs During Infusion |
|
| Any SAEs During Infusion |
|
| Any AEs Causing Study Discontinuation |
|
| Any Study Drug-Related AE Causing Study Discont. |
|
| AEs Causing Permanent Study Drug Discont. |
|
| Drug-Related AE Causing Permanent StudyDrug Discon |
|
| Any SAEs Causing Study Discontinuation |
|
| Any SAEs Causing Permanent Study Drug Discont. |
|
| Study Drug-Related SAE Causing Study Discont. |
|
| StudyDrug-Related SAE Causing Permanent DrugDiscon |
|
| Death |
|